The era of off-label guesswork for patients living with the rare autoimmune disorder MOGAD may soon be over, as a phase 3 ...

After a rare disease diagnosis left her partially paralyzed, Marion Jones worked her way back from being reliant on a ...

The traditional gold standard of clinical trials with a randomized, placebo-controlled design involving hundreds of patients ...

Rare diseases, defined as conditions that affect a small percentage of the population, represent a significant challenge in medical research and healthcare. Collectively, these diseases encompass a ...

Lozano is a rare disease mom, neuroscience Ph.D. candidate at UC Davis, and board member for the PURA Syndrome Foundation. In May, a historic moment in science and medicine was captured in a single ...

For most parents, a medical diagnosis can take minutes. But for one Florida family, it took years. In 2019, Jordan Avi Ogman ...

After receiving the FDA’s greenlight for Hunter syndrome drug Avlayah, Denali Therapeutics CEO Ryan Watts saw the culmination ...

Stecker is a rare disease mother and advocate and served as assistant secretary of public affairs and deputy chief of staff at the Department of Health and Human Services. Every parent dreams of a ...

GeneDx, which specializes in diagnostics for rare and ultra-rare genetic disorders, is helping to get kids the diagnostic attention they need as part of Diagnosis is Power. Currently, one in 10 ...

Our inbox has been flooded with emails from viewers and readers who told us how their lives have been affected by rare diseases. Here are some of the stories we received. Living with a rare disease ...

AstraZeneca AZN announced positive results from an interim analysis of the ongoing late-stage I CAN study, which evaluated ...

A UK man is honouring his late wife, Katy Miles, who died at 46 from a rare ovarian cancer. Her only early warning was an ...